Brief Summary

Dystonia is a movement disorder seen in both children and adults that is characterized by "sustained or intermittent muscle contractions causing abnormal, often repetitive, movements, postures, or both." Secondary dystonia is far more common in pediatric populations than primary dystonia, and far more recalcitrant to standard pharmacologic and surgical treatments including Deep Brain Stimulation (DBS). There exists a large unmet need to develop new therapeutics, treatment strategies, and outcome measures for pediatric secondary dystonia. The investigators are proposing to investigate the ventralis oralis posterior nucleus (Vop) of the thalamus as a new target for DBS in secondary dystonia. Prior to the development of DBS, the main surgical treatment of dystonia was thalamotomy. Although there were many different targets in the thalamus, often done in staged procedures, the most common and successful targeted nuclei was the Vop, which is traditionally thought to be the pallidal receiving area. Previous lesioning of Vop produced improvements in dystonia but intolerable side effects, especially when implanted bilaterally. However, given that secondary dystonia patients were often reported to have superior results to primary dystonia it is reasonable to believe that if the side effects can be modulated, that targeting of the Vop nucleus with DBS could be a viable alternative to Globus Pallidus interna (GPi). Given that Deep Brain Stimulation is a treatment that is inherently adjustable, it is conceivable that settings on the Deep Brain Stimulation could be adjusted to allow for clinical benefit with minimal side effects. Indeed, there have been several scattered successful case reports attesting to this possibility.

Trial Health

On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment

participants targeted

Target at below P25 for not_applicable

Timeline

Completed

Started Mar 2017

Typical duration for not_applicable

Geographic Reach

1 country

1 active site

Status

completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

2.4 years study duration

First Submitted

Initial submission to the registry

April 26, 2016

Completed

10 months until next milestone

Study Start

First participant enrolled

March 3, 2017

Completed

10 days until next milestone

First Posted

Study publicly available on registry

March 13, 2017

Completed

2.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 24, 2019

Completed

Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

July 24, 2019

Completed

1.2 years until next milestone

Results Posted

Study results publicly available

October 8, 2020

Completed

Last Updated

October 8, 2020

Status Verified

September 1, 2020

Enrollment Period

2.4 years

First QC Date

April 26, 2016

Results QC Date

July 21, 2020

Last Update Submit

September 14, 2020

Conditions

Dystonia

Outcome Measures

Primary Outcomes (4)

Change From Baseline in Burke-Fahn-Marsden Dystonia Rating Scale
Rating scale that measures movement and disability related to dystonia, range 0-120 motor, 0-30 disability , higher number indicates more severe dystonia Change from Baseline in Burke-Fahn-Marsden Dystonia Rating Scale
Change from baseline to 12 months postoperatively
Percent Change in Pediatric Quality of Life Inventory (PedsQL)
Quality of life measure, scored 0-100, larger scores indicate greater hinderance (ie. lower quality of life)
baseline to 12 months postoperatively
Change in Barry Albright Dystonia Rating Scale
Severity scale for secondary dystonia, range 0-32, higher scores indicates more severe dystonia
Change from baseline to 12 months postoperatively
Change in Blinded Burke-Fahn-Marsden Dystonia Rating Scale
Rating scale that measures movement and disability related to dystonia, range 0-120 motor, 0-30 disability , higher number indicates more severe dystonia. These ratings were carried out retroactively by a neurologist who was unfamiliar with the four study participants and who had no knowledge of their unblinded scores.
change from baseline to 12 months postoperatively

Secondary Outcomes (8)

Change in Modified Ashworth Scale - Upper Limbs
Change from baseline to 12 months postoperatively
Change in Diadochokinetic Syllable Rates
Change from baseline to 12 months postoperatively
Children's Memory Scale
Change from baseline to 12 months postoperatively
Change in Behavioral Assessment System, 3rd Edition: Self Report of Personality
Change from baseline to 12 months postoperatively
Change in Modified Ashworth Scale Spasticity Ratings - Lower Limbs
Change from baseline to 12 months postoperatively
+3 more secondary outcomes

Study Arms (1)

DBS active

EXPERIMENTAL

All participants will be enrolled in DBS placement and active stimulation. The following components will be used: * Activa PC Primary Cell Neurostimulator - (Model 37601) * Activa RC Rechargeable Neurostimulator - (Model 37612) * Activa SC Single Cell Neurostimulator (Models 37602 and 37603) * DBS Lead - (Model 3387) * DBS Extension - (Models 37085/6) * Patient Programmer - (Model 37642) * Test Stimulator - (Model 3625) * N'Vision Clinician Programmer - (Model 8840) * N'Vision Software Application Card - (Model 8870)

Device: Activa PC Primary Cell Neurostimulator - (Model 37601)Device: Activa RC Rechargeable Neurostimulator - (Model 37612)Device: Activa SC Single Cell Neurostimulator (Models 37602/37603)Device: DBS Lead - (Model 3387Device: DBS Extension - (Models 37085/6)Device: Patient Programmer - (Model 37642)Device: Test Stimulator - (Model 3625)Device: N'Vision Clinician Programmer - (Model 8840)Device: N'Vision Software Application Card - (Model 8870)